title: Combination of melphalan and dexamethasone for patients with newly diagnosed POEMS syndrome.
abstract: POEMS syndrome is a rare clonal plasma cell disorder without standard treatment. Based on the efficacy and low toxicity of a combination of melphalan and dexamethasone (MDex) for light chain amyloidosis, we conducted a prospective study of MDex treatment for patients with newly diagnosed POEMS syndrome. Thirty-one patients (19 men) were enrolled and the median age at the time of diagnosis was 44 years (range, 32-68 years). All patients received 12 cycles of MDex treatment. Twenty-five patients (80.6%) achieved hematologic response including 12 (38.7%) complete remission and 13 (41.9%) partial remission. Of all 31 patients, the neurologic response rate was 100%, assessed by overall neuropathy limitation scale (ONLS). The initial neurologic response was observed in 24 patients (77.4%) at 3 months after treatment and the median time to maximal neurologic response was 12 months (range, 3-15 months). Moreover, MDex substantially improved the level of serum vascular endothelial growth factor and relieved organomegaly, extravascular volume overload, and pulmonary hypertension. Only 6 patients (19.3%) suffered from grade 3 adverse events during treatment. All patients are alive and free of neurologic relapse after the median follow-up time of 21 months. Therefore, MDex is an effective and well-tolerated treatment option for patients with newly diagnosed POEMS syndrome.
authors.affiliation: Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, People’s Republic of China.
authors.forename: Jian
authors.initials: J
authors.lastname: Li
authors.forename: Wei
authors.initials: W
authors.lastname: Zhang
authors.forename: Li
authors.initials: L
authors.lastname: Jiao
authors.forename: Ming-Hui
authors.initials: MH
authors.lastname: Duan
authors.forename: Hong-Zhi
authors.initials: HZ
authors.lastname: Guan
authors.forename: Wei-Guo
authors.initials: WG
authors.lastname: Zhu
authors.forename: Zhuang
authors.initials: Z
authors.lastname: Tian
authors.forename: Dao-Bin
authors.initials: DB
authors.lastname: Zhou
mesh_terms: D000328:Adult; D017668:Age of Onset; D000368:Aged; D000893:Anti-Inflammatory Agents / Q000008:administration & dosage / Q000009:adverse effects; D003907:Dexamethasone / Q000008:administration & dosage* / Q000009:adverse effects; D004359:Drug Therapy, Combination; D005260:Female; D006801:Humans; D008297:Male; D008558:Melphalan / Q000008:administration & dosage* / Q000009:adverse effects; D008875:Middle Aged; D019653:Myeloablative Agonists / Q000008:administration & dosage / Q000009:adverse effects; D020360:Neoadjuvant Therapy; D016878:POEMS Syndrome / Q000175:diagnosis / Q000188:drug therapy* / Q000453:epidemiology / Q000473:pathology; D016896:Treatment Outcome
chemical_list: D000893:Anti-Inflammatory Agents; D019653:Myeloablative Agonists; D003907:Dexamethasone; D008558:Melphalan